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juvenile GM1 gangliosidosis

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Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients

Recruiting
  • GM1 Gangliosidosis
    • Oakland, California
    • +5 more
    Feb 2, 2023

    Interviews and Video Capture in Patients With GM1 Gangliosidosis

    Recruiting
    • GM1 Gangliosidosis
    • Natural history
    • Plymouth, Massachusetts
      Casimir Trials
    Aug 23, 2021

    GM1 and GM2 Gangliosidosis PROspective Neurological Disease

    Recruiting
    • GM1 Gangliosidosis
    • +2 more
      • Oakland, California
      • +12 more
      Nov 16, 2022

      Lysosomal Diseases, Gangliosidosis, GM1 Trial run by the NHGRI (biological, drug, diagnostic test, other, procedure, behavioral)

      Recruiting
      • Lysosomal Diseases
      • +2 more
      • AAV9-GLB1
      • +20 more
      • Bethesda, Maryland
        National Institutes of Health Clinical Center
      Jan 19, 2023

      A Natural History Study of the Gangliosidoses

      Recruiting
      • Tay-Sachs Disease
      • +4 more
        • Minneapolis, Minnesota
          University of Minnesota - Pediatric Genetics and Metabolism
        Feb 28, 2022

        GM1 Gangliosidosis, GM1 Gangliosidosis, Type I, GM1 Gangliosidosis, Type 2 Trial in Worldwide (PBGM01)

        Recruiting
        • GM1 Gangliosidosis
        • +3 more
        • PBGM01
        • Oakland, California
        • +9 more
        Mar 10, 2022

        GM2 Gangliosidosis, Niemann-Pick Disease, Type C Trial in Curitiba, Porto Alegre, Rio De Janeiro (AZ-3102 (Dose 1), Placebo,

        Not yet recruiting
        • GM2 Gangliosidosis
        • Niemann-Pick Disease, Type C
        • AZ-3102 (Dose 1)
        • +2 more
        • Curitiba, Brazil
        • +2 more
        Feb 24, 2023

        GM1 Gangliosidosis Trial in Orange, Paris, Manchester (LYS-GM101)

        Recruiting
        • GM1 Gangliosidosis
        • LYS-GM101
        • Orange, California
        • +2 more
        Jun 30, 2021

        Tay-Sachs Disease Sandhoff Disease Trial in Worldwide (venglustat GZ402671, )

        Active, not recruiting
        • Tay-Sachs Disease Sandhoff Disease
        • venglustat GZ402671
        • placebo
        • Los Angeles, California
        • +23 more
        Jan 31, 2022

        Infantile GM2 Gangliosidosis (Disorder) Trial in Kingston (TSHA-101)

        Active, not recruiting
        • Infantile GM2 Gangliosidosis (Disorder)
        • TSHA-101
        • Kingston, Ontario, Canada
          Queen's University/Kingston Health Sciences Centre
        Sep 12, 2022

        Brain Metastases, Radiotherapy Side Effect, Cognitive Impairment Trial in Guangzhou (Monosialotetrahexosyl ganglioside (GM1),

        Recruiting
        • Brain Metastases
        • +4 more
        • Monosialotetrahexosyl ganglioside (GM1)
        • Control
        • Guangzhou, Guangdong, China
          Sun Yat-sen University Cancer Center
        Nov 3, 2021

        Biomarker for Gangliosidosis: BioGM1/BioGM2 (BioGM1/GM2)

        Active, not recruiting
        • Hepato-splenomegaly
        • +3 more
          • Cairo, Egypt
          • +4 more
          May 12, 2021

          Glycosphingolipid and Glycoprotein Lysosomal Storage Disorders

          Recruiting
          • Neurological Regression
          • +3 more
            • Bethesda, Maryland
              National Institutes of Health Clinical Center
            Jan 14, 2023

            GM1 Gangliosidoses, GM2 Gangliosidoses, Tay-Sachs Disease Trial in Minneapolis (miglustat, Ketogenic Diet)

            Terminated
            Has Results
            • GM1 Gangliosidoses
            • +3 more
            • Minneapolis, Minnesota
              University of Minnesota
            Mar 18, 2021

            Gangliosidoses, GM1 Gangliosidosis, Morquio B Disease Trial in Hochheim

            Recruiting
            • Gangliosidoses
            • +8 more
              • Hochheim, HE, Germany
                SphinCS Lyso gemeinnützige UG (haftungsbeschränkt)
              Nov 5, 2020

              The Natural History of Metachromatic Leukodystrophy

              Recruiting
              • MLD
              • +26 more
              • Palliative Care
              • Hematopoetic Stem Cell Transplantation
              • Pittsburgh, Pennsylvania
                The Program for the Study of Neurodevelopment in Rare Disorders,
              Nov 2, 2021

              ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn

              Enrolling by invitation
              • Acid Sphingomyelinase Deficiency
              • +13 more
              • Confirmatory Testing
              • Bronx, New York
              • +9 more
              May 9, 2022

              GM2 Gangliosidosis, Supportive Care Trial in Kashan, Mashhad, Tehran (Miglustat)

              Recruiting
              • GM2 Gangliosidosis
              • Supportive Care
              • Kashan, Isfahan, Iran, Islamic Republic of
              • +2 more
              Oct 23, 2022

              Tay-Sachs Disease, Sandhoff Disease Trial in Boston, Worcester (AXO-AAV-GM2 Starting Dose, AXO-AAV-GM2 Low Dose, AXO-AAV-GM2

              Recruiting
              • Tay-Sachs Disease
              • Sandhoff Disease
              • AXO-AAV-GM2 Starting Dose
              • +3 more
              • Boston, Massachusetts
              • +1 more
              Jan 26, 2021

              Juvenile Idiopathic Arthritis Trial in Izmir (Inspiratory muscle training (IMT))

              Enrolling by invitation
              • Juvenile Idiopathic Arthritis
              • Inspiratory muscle training (IMT)
              • İzmir, Turkey
                Izmir Katip Celebi University
              Jul 29, 2022

              To Evaluate the Effectiveness of GM1 in the Prevention of Albumin-paclitaxel Therapy Induced Peripheral Neuropathy in Breast

              Not yet recruiting
              • To Evaluate the Effectiveness of GM1 in the Prevention of Albumin-paclitaxel Therapy Induced Peripheral Neuropathy in Breast Cancer Patients
              • Monosialotetrahexose ganglioside sodium injection
              • (no location specified)
              Mar 27, 2023

              Anatomic Stage IV Breast Cancer AJCC v8, Chemo-Induced Peripheral Neuropathy, Metastatic Breast Carcinoma Trial (Paclitaxel,

              Not yet recruiting
              • Anatomic Stage IV Breast Cancer AJCC v8
              • +2 more
              • (no location specified)
              Feb 21, 2023

              Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy Trial in Minneapolis (drug, procedure,

              Completed
              Has Results
              • Adrenoleukodystrophy
              • +8 more
              • Minneapolis, Minnesota
                Masonic Cancer Center, University of Minnesota
              Jul 10, 2019

              Opioid Use, Substance Use Trial in United States (Trust-based Relational Intervention Group Training, Trust-based Relational

              Recruiting
              • Opioid Use
              • Substance Use
              • Trust-based Relational Intervention Training
              • +2 more
              • Chicago, Illinois
              • +11 more
              Jan 20, 2023

              Juvenile Systemic Lupus Erythematosus

              Recruiting
              • Systemic Lupus Erythematosus
              • lipid profile
              • Assiut, Egypt
                Assiut university
              Nov 21, 2023